Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients

Chronic Granulomatous Disease Clinical Trial

Official title:

A Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03547830
• Other study ID #: NCPHOI-2018-02
• Status: Recruiting
• Phase: Phase 2
• Start date: April 13, 2019
• Est. completion date: January 1, 2023

Study information

• Verified date: September 2019
• Source: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
• Contact: Dmitry Balashov, MD
• Phone: +79265791817
• Email: bala8[@]yandex.ru
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation in patients with chronic granulomatous disease

Description:

Severe primary or secondary graft dysfunction is one of major problem in patients with Chronic granulomatous disease (CGD). In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages. The effect is based on mobilizing bone marrow stem cells into the peripheral blood and blocking CXCR4 chemokine receptors to prevent stem cell homing. Thus, some have hypothesized that plerixafor and G-CSF make free stromal space of the bone marrow available for donor stem cell engraftment. Moreover, stem cell release probably leads to liberation of host stem cells from the anti-apoptotic effects of the BM stroma for the more powerful effect of chemotherapy. Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after stem cell transplantation in patients with chronic granulomatous disease.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 17
• Est. completion date: January 1, 2023
• Est. primary completion date: January 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 24 Years

Eligibility

Inclusion Criteria:

Patients aged = 1 months and < 25 years Patients diagnosed with CGD eligible for an allogeneic transplantation Signed written informed consent

Exclusion Criteria:

Lack of informed consent.

Related Conditions & MeSH terms

• Chronic Granulomatous Disease
• Granuloma
• Granulomatous Disease, Chronic

Intervention

Drug:
• Plerixafor
Plerixafor for Conditioning before HSCT.
• Gcsf
GCSF for Conditioning before HSCT.

Locations

Country	Name	City	State
Russian Federation	Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology	Moscow

Sponsors (1)

Lead Sponsor	Collaborator
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Country where clinical trial is conducted

Russian Federation, 

References & Publications (1)

Balashov D, Laberko A, Shcherbina A, Trakhtman P, Abramov D, Gutovskaya E, Kozlovskaya S, Shelikhova L, Novichkova G, Maschan M, Rumiantsev A, Maschan A. A Conditioning Regimen with Plerixafor Is Safe and Improves the Outcome of TCRaß(+) and CD19(+) Cell-Depleted Stem Cell Transplantation in Patients with Wiskott-Aldrich Syndrome. Biol Blood Marrow Transplant. 2018 Jul;24(7):1432-1440. doi: 10.1016/j.bbmt.2018.03.006. Epub 2018 Mar 14. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Event free survival	The EFS probability compared with historical control. We mean event as primary (non-engraftment) and secondary (rejection) graft dysfunction.	1 year
Secondary	1. Overall survival	The OS probability compared with historical control	1 year
Secondary	Proportion of patients with full/mixed donor chimerism	Evaluation of the percentage of patients with the full/mixed donor chimerism (whole blood and CD3+ lineage). In addition, patients will be divided in accordance with % of donors cells: >95%; 50%-95%; 10%-49%; <10%. All data will be compared with historical control	30 days
Secondary	3. Transplant related mortality	The TRM probability compared with historical control.	1 year
Secondary	4. Acute Graft Versus Host Diseases	Cumulative Incidence of aGVHD	100 days
Secondary	5. Incidence of Plerixafor related toxicity	severity, features, incidence	100 days